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This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.
Full description
γ-globin reactivated autologous hematopoietic stem cells will be manufactured using Glycosylase Base Editors. Subject participation for this study will be 2 year. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 15 years post-transplant.
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Inclusion and exclusion criteria
Key inclusion criteria:
Fully understand and voluntarily sign informed consent. 3-35years old. At least one legal guardian and/or Subjects to sign informed consent.
Clinically diagnosed as β-thalassemia major, phenotypes including β0β0, β+β+、β
+β0, βEβ0 genotype.
Subjects with no affection with EBV, HIV, CMV, TP, HAV, HBV and HCV.
Subjects body condition eligible for autologous stem cell transplant.
Key exclusion criteria:
Primary purpose
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Interventional model
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5 participants in 1 patient group
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Central trial contact
lai yongrong, PhD; LI WEI, PhD
Data sourced from clinicaltrials.gov
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